Focusing on Value as Defined by Patients

In times of constrained resources, access to new treatments can benefit patients, the health care system and the economy.

To keep progress in cancer research and care moving forward, policymakers must not lose sight of patient needs and preferences, a new report says.

In a special report published in the journal Clinical Cancer Research, the Turning the Tide Against Cancer Through Sustained Medical Innovation coalition outlines some of the challenges that innovation in cancer research faces today. To support innovation, the authors conclude that a new perspective in cancer research is needed, one that emphasizes patient-centered value.

“We foresee that with continued support for science-based innovation, we can accomplish in oncology what has been achieved against other major public health problems, such as HIV/AIDS, where scientific advances yielded major gains for patients and averted a predicted health spending crisis,” said study author Zeba Khan, an advisory panel member of the Turning the Tide coalition and vice president of Global Strategic Market Access and Policy at Celgene.

One major challenge facing cancer research is the different views on how we judge medical innovation. For example, in a 2008 survey conducted by Zitter Health Insights, over two-thirds of cancer researchers said that a therapy has a survival benefit if it extends a patient’s life by three to six months. In contrast, those who help pay for care—such as insurance companies and health plan sponsors—said that figure should be more than nine months.

“Many payers and policymakers understandably want assurance that continued progress will mean greater efficiency and higher value in cancer care,” Khan said.

It’s vital that stakeholders continue to discuss and advocate for policy changes that will enable scientific advances to improve outcomes for patients.

But these differing perceptions suggest that we need to rethink how we define and assess value, according to the authors of the Turning the Tide report.

Clinical value is a good starting place to develop a more collective definition, the authors say. This definition includes cost and effectiveness as well as quality of life, productivity and patient preference. This definition emphasizes not only how long patients live but also how they feel during their treatment.

But current value assessment approaches—such as comparative effectiveness research and cost-effectiveness analysis—does not provide a complete picture of value, according to the report. These strategies do not keep pace with science or take into account the varying needs of patients as well as the positive impact that medical innovation has on the health care system and the economy.

For example, if the UK’s National Institute for Health and Clinical Excellence (NICE) adopted a more value-based model that considers the benefits of new medications to patients, health care and the economy, cancer patients in that country today could have access to new therapies for up to 226 additional indications.

A complete picture of effectiveness and value only comes when a treatment enters real world use, wrote the authors of the Turning the Tide report. Over time, patients taking new treatments may show continued signs of improvement in incremental steps that add up to larger, highly meaningful differences—as happened in the case of HIV treatments. In addition, therapies might even show benefits for other cancers than the one for which they were initially approved. By following patients after their course of treatment, new favorable outcomes can be identified for therapies long after their participation in a trial.

Right now, several groups are working to implement the strategies outlines by the coalition. At the policy level, the Personalized Medicine Coalition is defining policy reforms that will provide incentives for innovation that focus on patient needs. At the patient level, a national network called the Patient-Centered Outcomes Research Institute is fostering shared patient, physician and caregiver decision-making.

“There is growing support across the cancer community for policies that align with and support continued progress,” Khan said. “It’s vital that stakeholders continue to discuss and advocate for policy changes that will enable scientific advances to improve outcomes for patients.”